FAQs About Our Clinical Trial

What are the eligibility criteria for the CHM gene therapy trial?

I have choroideremia and would like to be considered for this trial. Can I be put on the list?

How many study participants do you expect to recruit for the trial?

I do not live in Edmonton, Alberta. Would I still be eligible for clinical trial entry if I meet the criteria?

What will it cost me to participate in the clinical trial?

How long will it take to complete this Phase 1 gene therapy clinical trial?

Are there any other possibilities for me if I’m not selected to be part of the clinical trial?

Are there other researchers studying gene therapy for choroideremia?

Where do I get information on other clinical trials that are planned or may be recruiting?

Why is genetic testing necessary for gene therapy clinical trial candidates?

Are there any dietary recommendations for people who have choroideremia?

 

What are the eligibility criteria for the CHM gene therapy trial?

The criteria for inclusion in the clinical trial will be determined and clearly communicated before recruitment begins. Potential participants will be screened to see if they meet the inclusion criteria. Selection will depend partly on the preliminary results of patients having undergone gene therapy in Oxford, England. In addition, it will be limited to males, over age 18 and with a diagnosis of choroideremia confirmed by genetic testing. Baseline visual exams will also help determine patient eligibility. Generally speaking, a candidate’s symptomatic progression, genetic test result, as well as availability for the clinical trial and follow-up, vision monitoring visits will all be considerations for eligibility. Only Canadians who meet criteria will be recruited.

 I have choroideremia and would like to be considered for the gene therapy trial. Can I be put on the list?

Please contact us if you are interested in participating in this trial. This website will be updated regularly with the status of this study.

You may want to consider being a part of the Foundation Fighting Blindness Canada patient registry. Scientists who are studying retinal eye disease are encouraged to use this registry to find Canadian participants for their research. There are different goals in research. Some scientists are interested to learn about how retinal diseases develop, others are studying better ways to diagnose retinal diseases and still others are interested in finding a way to treat these conditions.

Our team is interested in finding a treatment for choroideremia. We may use this registry to identify and contact eligible individuals with choroideremia to recruit for future clinical trials. For more information on the patient registry, please click here.

How many study participants do you expect to recruit for the trial?

We will recruit twelve participants who meet the criteria.

I do not live in Edmonton, Alberta. Would I still be eligible for clinical trial entry if I meet the criteria?

It is possible that you may still be considered if you are Canadian and live elsewhere in the country.

What will it cost me to participate in the clinical trial?

Although visual assessments that are part of the clinical trial are covered, we currently do not have the funds to compensate you for your time, accommodation, or travel expenses.

How long will it take to complete this Phase 1 gene therapy clinical trial?

Each study participant will be followed closely for two years after gene therapy is administered. However, we do not expect to recruit all 12 study participants at the same time. In total, this trial will take five years.

Are there any other possibilities for me if I’m not selected to be part of the clinical trial?

Our study will begin with a relatively small group of participants. If this gene therapy is proven to be safe, we may move on to a larger clinical trial. However, at present, we only have funding for a small Phase 1 clinical trial. Unfortunately, we are unable to provide an estimated time frame for expanding the study.

Are there other researchers studying gene therapy for choroideremia?

Researchers in both England and the United States are also studying gene therapy for choroideremia. Dr. Robert MacLaren is leading the clinical trial that is taking place in England. For more information about this clinical trial, please click here. In the US, Drs. Jean Bennett and Albert Maguire are in the process of setting up a similar study in Pennsylvania.

Where do I get information on other clinical trials that are planned or may be recruiting?

Please refer to the ClinicalTrials.org website. Search for “choroideremia” for an up-to-date listing of clinical trials.

Why is genetic testing necessary for gene therapy clinical trial candidates?

Gene therapy is gene specific. Having a confirmed genetic mutation ensures that the gene therapy is targeting the correct gene in an affected individual. The gene that causes choroideremia is called CHM.

Where can I get a genetic test for choroideremia?

We provide genetic testing for CHM on a research basis. Please see the genotype-phenotype webpage for more information.

Are there any specific dietary recommendations for people who have choroideremia?

A balanced diet that includes omega-3 fatty acids and anti-oxidants is important for eye health. These recommendations are helpful to everyone.